In June 2011, medical regulators gave the go-ahead for trials in humans of a potential new anti-HIV drug. The drug was produced in genetically modified tobacco leaves. It was the first ever clinical trial of a drug of this type that had been derived from GM plants.
The hope is that the drug will prove effective in preventing HIV infection. But the real significance of the regulatory green light, given by the UK's licensing body, the Medicines and Healthcare products Regulatory Agency (MHRA), went further than that.
It confirmed, for the first time, that molecules known as monoclonal antibodies - the key component of the drug, and of many other highly effective modern pharmaceuticals - could be produced from plants in a form that met the extremely stringent standards required for use in the treatment of humans. As well as bringing hope to the fight against HIV/AIDS, the decision opened the way for trials of other plant-derived medicines to treat a range of diseases.
It was, potentially, an important step towards the transformation of modern drug manufacturing, offering the developing world access to key drugs which have previously been prohibitively costly.
The move to Phase 1 clinical trials was the crowning achievement of PHARMA-PLANTA, a seven-and-a-half year EU-funded project set up in 2004. With € 12 million of funding provided under the EU's 6th Framework Programme of research and development, the PHARMA-PLANTA consortium comprised more than 30 academic and industrial partners from across Europe and South Africa. The consortium's goal was clearly stated: to develop a manufacturing process for recombinant protein drug products derived from GM plants and to take one such product through all the development stages, including clinical trial.
The approval from the MHRA was described by the project's scientific co-ordinator, Professor Julian Ma of St. George's, University of London, as "a red letter day". It was, he said, "an acknowledgement that monoclonal antibodies can be made in plants to the same quality as those made using existing conventional production systems. That is something many people did not believe could be achieved."
The conventional production systems referred to by Professor Ma use sophisticated stainless steel fermentation vats containing bacteria or mammalian cells. The process of deriving antibodies from these cells is high-tech - and highly expensive.
By contrast, the antibodies produced by PHARMA-PLANTA were derived from tobacco plants grown in greenhouses in Germany, harvested after 45 days and shredded. According to Professor Rainer Fischer, Director of the institute where the GM tobacco was grown, this much simpler, more low-tech option is between 10 and 100 times cheaper.
Just as importantly, the simplicity of the process means it could easily be transferred to developing countries, allowing production of drugs "in the region, for the region."
Discovered by one of the four private commercial partners in the project, Austrian biotech company Polymun, the new antibody successfully completed its Phase 1 trial, ascertaining its safety, at the end of 2011. It is now set to move into the next phase of testing, to establish its medical effectiveness.
Meanwhile, other diseases for which it is envisaged that GM plants could provide new drugs include cancer, rheumatoid arthritis and others which, in the words of Professor Ma, are currently "horribly expensive" to treat.
As Professor Fischer explains, the success of PHARMA-PLANTA "is a springboard for European plant biotechnology and will enable many important medical products to be realised".
Participants The Netherlands, Germany (Coordinator), France, Italy, Belgium, Spain, Sweden, United Kingdom, Austria, Greece, Switzerland, South Africa
FP6 Proj. N° 503565
Total costs: € 17 600 000
EU contribution: € 12 000 000
Duration: from: Feb. 2004 to: Oct. 2011